Stem Cell Gene Therapy Essay - 903 Words

In recent years, hematopoietic stem cell gene therapy (HSC GT) has emerged as an innovative strategy to cure several primary immunodeficiency disorders such as Severe Combined Immunodeficiency (SCID) and Wiskott-Aldrich Syndrome (WAS). The Wiskott-Aldrich syndrome is an â€Å"X-linked, complex primary immunodeficiency disorder caused by mutations in the WAS gene and is characterized by recurrent infections, thrombocytopenia, eczema, autoimmunity and an increased risk of lymphoma† (1). Severe WAS can lead to fatality in the patient from infection or hemorrhage (1). Earlier, hematopoietic stem cell transplantation (HSCT) was used as the standard approach for the treatment of WAS but recently, due to the promising and long term results, HSC gene†¦show more content†¦Braun and his colleagues also performed a comprehensive analysis of the gamma-retroviral vector’s integration sites (IS) and concluded that the integrations were mostly accumulated at the transcription start site (TSS) of genes (1). The surprising element of the study was that most of these genes were known proto-oncogenes (like LMO2 and MDS1), indicating that the -retroviral vectors clearly has a preference towards oncogenes or proto-oncogenes for its integration and should thus be reconsidered for its use as a safe delivery system for gene therapy. In this study, the use of gamma-retroviral vector was associated with an exceptionally high rate of secondary malignancies in WAS, raising considerable safety concerns (2). This shortcoming has been increasingly raising concerns in researchers working on gene therapy and has been keeping them from using the readily available and easily transducible retroviral vectors for their experiments and trials. This is also the reason why most studies today use lentiviral vectors, which do not have the issue of causing insertional mutations, rather than retroviral vectors. To overcome the drawback of classic -retroviral vectors mentioned above, the authors of this study proposed the use of self-inactivating (SIN) viruses as vectors for gene therapy. A SIN retrovirus isShow MoreRelatedEssay On 20 Years Of Advancement In Healthcare Science1109 Words   |  5 Pageshealthcare science. These have allowed for improved diagnoses and more effective treatments lowering the prevalence of diseases. 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